The film industry has an old maxim that you should never work with animals or children.

Clearly, the pharmaceutical industry is well used to working with animals despite the problems that work brings, but drug companies have followed the actors’ maxim when it comes to children.

It is a remarkable and until recently little discussed fact that most of the drugs used on our children have not been tested specifically on this peer group for efficacy or safety. This means that when a doctor or other prescriber reaches for his or her prescription pad to relieve or remove the suffering of someone under 18, he or she is often effectively gambling with their career.

Maybe that is why the issue has had such a low profile - the professional and legal consequences for health professionals could be so huge that a panic could have massive short-term consequences with children denied treatment.

This historic anomaly is finally being addressed through a series of initiatives and regulatory changes.

While the principal beneficiaries will be prescribers and their patients, the changes could also open the way for new marketing tools.

Central to the new framework is a European Union regulation driven through under the UK presidency of the union last year. Supporters say the new regulation will broaden the range of medicines specifically adapted and licensed for use with children and provide health professionals, patients and parents with much-needed information about those drugs and other medicines used on children. According to the Medicines and Healthcare Products Regulatory Agency (MHRA), new information should start to flow soon after the regulation comes into force “by the end of 2006”.

In framing the regulation, EU policymakers have been careful to emphasise the carrot rather than the stick. Companies will be rewarded with an extra six months patent protection in return for carrying out the paediatric research – an incentive which has inevitably caused angst in the generics sector. Once the paediatric data has been collected and verified, those marketing the drug to health professionals will be able to point to a “P” for paediatric logo which the product will display.

The extended patient protection suggests that regulators have accepted that the current problems have arisen at least partly because emotion and natural parental concern have made recruitment for trials difficult. Previously, many firms have considered that the economics of expensive and lengthy research into a new product’s suitability for children do not add up - obviously there are fewer children than adults and therefore fewer potential customers. Additionally, the term “paediatric” covers a huge range of child development - anything from babies and infants to strapping 6ft teenagers.

However most in the industry have welcomed the move but also cautioned about the scale of the challenge facing drugs companies if hopes for the regulation are to be fulfilled.

Marie Manley, partner in the Healthcare Regulatory Practice at law firm Bristows, says: “The idea of drugs being tested and assessed for those who use them is obviously a good thing. The downside for the industry is that research and testing is very difficult and expensive especially for children. As a parent I think the idea is very good but at the same time I wouldn’t accept that my children should be involved. That situation is likely to be made worse by the recent case (at Northwick Park Hospital). As a lawyer I see this could be a very serious and difficult process that might delay the access of new drugs to the market.”

Ms Manley says there is acceptance that the status quo is fraught with difficulty.

“The guidance on prescribing out there is very helpful and I thank God it is there but the regulatory reality is that prescribers are in violation of the law a lot of the time.”

And regulators have signalled they intend to show some flexibility. Clearly, drugs for conditions which are never or rarely fond in children and young people - such as dementia - will be granted a waiver from the regulation. In other cases drugs might be granted a temporary licence if a company can show that the paediatric research would prevent adults gaining access to a beneficial new drug.

The avalanche of information which we can expect to emerge from the paediatric trials will doubtless find its way into another recent initiative aimed at improving the lot of prescribers and their juvenile patients, a paediatric version of the prescribing bible the British National Formulary.

The separate edition of the BNF was actually a plank in a Department of Health paediatric medicines strategy in 2004 which also outlined plans for the EU regulation.

UK Health Minister Lord Warner said: “Health professionals need the latest information so that they can make the right choices abut the medicines and treatments for their younger patients and that is why the new BNF for children is so important.”

It is just possible that with new data and the special edition of the BNF, health professionals and the industry might just have the script which makes working with children a lot less painful than those entertainment luvvies make out.

What the new law means:

  • For new products, a requirement for paediatric data based on a paediatric investigation plan (PIP) unless they have a waiver or deferral;
  • an extra six months of protection of patent if that data is included in the final Summary of Product Characteristics (SmPC);
  • for “orphan” medicinal products, a two year extension of market exclusivity if data from a PIP is included in the SmPC; and
  • for off-patent products, a ”paediatric use marketing authorisation” or PUMA, will be introduced to provide market protection for ten years.

By Chris Mahoney